One-Shot gene therapy aims to free patients from lifelong blood transfusions
Disease control
Not yet recruiting
This study is testing a single injection of a patient's own genetically modified cells to treat severe beta-thalassemia. The goal is to see if this one-time treatment is safe and can help patients produce enough of their own healthy hemoglobin to stop needing regular blood transf…
Phase: PHASE2 • Sponsor: CorrectSequence Therapeutics Co., Ltd • Aim: Disease control
Last updated Apr 01, 2026 14:41 UTC